Participation in a clinical trial can provide access to cutting-edge, potentially lifesaving and life-enhancing treatments.
A clinical trial is a research study that helps to determine whether a new drug or device is safe and effective. Each trial is designed to answer questions and find better ways to screen, diagnose, prevent or treat a disease or condition. They may be sponsored by drug manufacturers, government, and organisations including the Leukaemia Foundation.
What happens during a clinical trial?
Each trial has a plan that maps out the study–what will be done, by whom, when and why. This protocol also explains who is eligible to participate in a trial and what is expected of them. If you are eligible, a team of doctors and nurses will manage your care. Trials are held at hospitals and research centres around the country.
What are the benefits of participating in a clinical trial? Should I consider taking part?
Participation in a trial gives you access to cutting-edge, potentially life-saving and life-enhancing treatments. Your participation contributes to the advancement of medicine and helps others who share or may develop your condition.
Before signing up, learn as much as possible about the trial, then discuss your options with your doctor. Trials are not right for everyone, nor is every patient able to participate. Before starting any trial, you will be given a Patient Informed Consent document to read, which includes a schedule of events and explains what will happen during the trial, what is expected of you, the care you will receive and the costs that you may have to cover. You will then be given a consent form to sign. Ask your doctor questions and be sure you have read and understand all aspects before you sign the Informed Consent document.
What will the cost be?
You will not need to pay any money to participate in a trial, over and above what you may pay for standard treatment. Any medical tests and medical care related to the trial are also at no cost. Some studies compensate you for other expenses such as parking and travel. Private insurers often do not cover the costs related to a trial, so you will need to check with your health care provider.
What are the risks – isn’t it dangerous to take an experimental drug?
The risks depend on the type of treatment being studied and the health of the individual patient. For some, there could be unpleasant, even serious, side effects. Often these side effects are temporary and end when the treatment stops. There are both known and unknown risks with any trial. Be sure you understand the known risks before you join any trial.
Whilst most trials involve some risk, researchers must follow strict scientific guidelines and ethical and legal codes to ensure that you are protected. Studies need to be approved by an Independent Ethics Committee (IEC) or Institutional Review Board (IRB), or an equivalent, depending upon the regulations of the country where the trial is being carried out.
This committee – made up of scientists, doctors, and other people from the local community (e.g., consumer representative, ethics expert) – reviews each trial to see that it is designed to protect the patient and to ensure that the benefits of the trial outweigh the risks. Each trial must meet the Good Clinical Practice (GCP) standard. GCP is an ethical and scientific quality standard that ensures that the rights, safety and well being of trial participants are protected.
How long will the clinical trial last?
The length of each trial is different. The trial’s protocol document will provide you with information on how long the trial lasts and what is expected of you.
You can leave the trial at any time. You can withdraw your consent and choose to leave the trial if you no longer wish to participate for any reason.
Will I still get regular medical care?
As a participant of a trial, you would receive excellent medical care from a team of doctors, nurses, researchers, social workers and other health professionals who are on hand to manage your condition. The trial’s protocol may require you to visit the trial site more often to check in with your trial doctor. Plus you may receive more tests and treatments than usual.
What if I get a placebo?
A placebo is a substance that has no therapeutic effect, and is given as a control when testing new medications. As a rule, trials of drugs for cancer do not use a placebo but rather participants receive a standard treatment already in use or the standard treatment plus the drug being studied. In “randomised” trials, researchers use a computer to randomly decide who will get the experimental treatment and who will receive the standard treatment. In a “blinded” trial, neither the researchers nor you will know if you’re receiving the experimental or standard drug. The randomised system ensures the process meets the scientific requirements of the trial.
Does my doctor have to be one of the doctors involved in the trial for me to take part?
No, your doctor does not have to participate in the trial in order for you to join. Trial researchers may provide you with care or they will want your regular doctor to care for you. Whether or not your doctor participates in the trial, you will need to see them for general medical care. Sometimes you may have to attend a different treatment centre and have a new doctor to participate in a trial.
How do I find out if I’m eligible?
Each trial protocol has guidelines stating who can and cannot join. The criteria vary by trial and could include your age, gender, medical history, current health status and the particular type or stage of disease you may have. Before you join the trial, you will be asked to sign an informed consent form. Then a doctor or nurse will assess your medical history, perform a physical exam and perform laboratory tests to determine whether you meet the eligibility criteria.
Is everyone with my disease eligible?
No, only people meeting the trial’s guidelines, or eligibility criteria, may join the trial. If you are found to be ineligible, you should talk to your doctor to see if there is another trial that may be right for you.
What would be required of me if I participate?
The doctor will first talk to you about informed consent. This is a process by which you will learn the details of the trial – what is involved, the purpose of the trial, the tests and procedures that will be used, and the risks and benefits. You will then be given a written consent form, which explains the trial. If you agree to take part, you will be asked to sign the form. If there is something on the form you do not understand, ask questions. Trial doctors and nurses are available to help you understand the risks and benefits of the trial.
What happens at the end of the trial – will I still be able to receive the drug?
After you complete the trial, you may or may not be able to continue receiving the drug. In some cases the treatment will not be made available to you again until it is government-approved. Once the trial ends, researchers analyse the data and if the trial is considered pivotal and results are positive they will be submitted to the national health authority for approval. During the approval process some pharmaceutical companies choose to continue to make the drug available through a pre-approval access program.
How do I find a clinical trial?
First, talk to your doctor. They will be able to access an up-to-date listing of trials. You may also want to call patient advocacy groups and local university medical centres. The Australasian Leukaemia & Lymphoma Group lists on its website currently open trials in blood and bone marrow cancers including leukaemia, lymphoma and myeloma. Other Australian trials sites include:
- Australian Cancer Trials – a website funded by Cancer Australia for those who have cancer and are considering taking part in a trial to test a new treatment.
- Australasian Leukaemia & Lymphoma Group (ALLG) – an organisation that specialises in clinical research, both through clinical trials and applied laboratory research associated with trials, for patients with a blood cancer.
- The Australian New Zealand Clinical Trials Registry – an online register of clinical trials being undertaken in Australia, New Zealand and elsewhere. A search function allows you to conduct a basic or advanced search of clinical trials.
- ClinTrial Refer – a mobile app and website platform that allows patients to independently search for actively recruiting clinical trials and to access trial site locations and contact details in real time.
- NHMRC Clinical Trials Centre – does not recruit participants to clinical trials directly, rather, it coordinates large multicentre clinical trials in Australia and promotes high quality clinical trials research.
Where can I get more information about clinical trials?
- ABC Health Matters website – Clinical Trials – a good vernacular description, and common questions.
- Therapeutic Goods Administration (TGA) website – includes ‘at a glance’ information and a list of frequently asked questions.
- World Health Organization
Clinical trials overseas
- ClinicalTrials.com (US)
- ClinicalTrials.gov (US)
- Cancer Trial Treatment Options
- National Cancer Institute (US)
Understanding clinical trials
In this webinar, facilitated by the Leukaemia Foundation in 2022, we discuss the what, why, and how of clinical trials to help you make informed decisions.
More information about the ClinTrial Refer App
A/Professor Nada Hamad, haematologist at St Vincent’s Hospital in Sydney, shares the benefits of the ClinTrial Refer mobile app.
Warren’s clinical trial story
Warren’s story isn’t unusual – prior to his 2003 diagnosis, he had no awareness of blood cancer, let alone chronic lymphocytic leukaemia (CLL). After seven rounds of chemotherapy, a bone marrow biopsy indicated he was running out of options. Until he learned of a targeted oral therapy overseas. The therapy was brought to Australia as part of a clinical trial and Warren was fortunate enough to be able to access the treatment that has kept him alive.